trofinetide
Trade name: Daybue
PeptideapprovedOrphan Drug FDAFast Track FDA
Approved
Mar 10, 2023
Trofinetide, sold under the brand name Daybue, is a medication used for the treatment of Rett syndrome. It is taken by mouth. — Wikipedia
Clinical trial activity
4 trials · 1 clinical orgs · 1 marketing orgs
Phase 1
0
Phase 2
3
Phase 3
4
Phase 4
0
Earliest trial started Nov 6, 2019 (NCT04181723)
Timeline
2010s
- Feb 11, 2015
Orphan Drug Designation
- Jan 1, 2019
Acadia Pharmaceuticals — Earliest Phase 3 Sponsor(trial)
2020s
- Jan 1, 2021
Acadia Pharmaceuticals — Earliest Phase 2 Sponsor(trial)
- Mar 10, 2023
Acadia Pharmaceuticals — Earliest FDA Approval
- Mar 10, 2023
Acadia Pharmaceuticals — Marketing Organization
- Mar 10, 2023
Acadia Pharmaceuticals — NDA Secondary Org
- Mar 10, 2023
Acadia Pharmaceuticals — NDA Organization
Indications
Approved for
Studied for
Mechanism of action
- Unspecified targetOTHER
Unknown
- Unspecified targetSupplementation
supplementation
Approval history
- approvedPriority reviewFast trackMar 10, 2023
Chemistry & pharmacology
Loading structure…
SMILES
C[C@@]1(C(=O)N[C@@H](CCC(=O)O)C(=O)O)CCCN1C(=O)CN- Mol. weight
- 315.33 g/mol
- Lipinski Ro5
- Pass
- Rule of 3
- No
- Chirality
- Single Stereoisomer
- Inorganic
- No
- Polymer
- No
- Delivery
- Oral
- Availability
- Prescription Only
- Multi-specific
- No
Oral
Yes
Parenteral
No
Topical
No
First in class
Sources
- WikipediaTrofinetide ↗
- ChEMBLCHEMBL197084 ↗
Clinical trials
| NCT ID | Phase | Start date | Sponsor(s) |
|---|---|---|---|
| NCT04988867 | Phase 2/Phase 3 (Phase 3) | Sep 22, 2021 | Acadia Pharmaceuticals |
| NCT04776746 | Phase 3 | Nov 8, 2020 | Acadia Pharmaceuticals |
| NCT04279314 | Phase 3 | Jan 29, 2020 | Acadia Pharmaceuticals |
| NCT04181723 | Phase 3 | Nov 6, 2019 | Acadia Pharmaceuticals |
Showing 4 of 4 trials
Page 1 / 1
Organizations
Research & Development (1)
| Organization | Org type | Trials | As lead sponsor | Phases | Earliest year |
|---|---|---|---|---|---|
| Acadia Pharmaceuticals | For profit | 4 | 4 | 1 | 2019 |
1 organizations
Page 1 / 1
Marketing (3)
| Organization | Org type | Relationship | Date |
|---|---|---|---|
| Acadia Pharmaceuticals | For profit | MKTG | Mar 10, 2023 |
| Acadia Pharmaceuticals | For profit | NDA2 | Mar 10, 2023 |
| Acadia Pharmaceuticals | For profit | NDA | Mar 10, 2023 |